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Sarepta Trial Protocol Changes May Ignite Debate On Placebo Design

Executive Summary

US FDA’s external advisors will meet May 18 to consider allowing use of central venous access lines in a study of two Duchenne muscular dystrophy drugs. Agency briefing documents show decision-making on placebo-controlled trial design was informed by problems in the clinical development of Exondys 51 (eteplirsen).

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US agency’s experts unanimously support allowing use of in-dwelling ports to aid infusions in trial of two Duchenne muscular dystrophy compounds, while also endorsing the two-year, double-blind, placebo-controlled design, which was influenced by the clinical experience with Exondys 51 (eteplirsen).

Eteplirsen Revisited? FDA Panel To Weigh Protocol Changes For Two Sarepta Drugs

US agency’s advisory committees will consider whether ongoing trial of exon 45- and 53-skipping compounds for Duchenne muscular dystrophy should allow use of in-dwelling ports to aid infusion.

Eteplirsen Review Offers Lessons For FDA, Advocacy Groups, Industry

Agency reviewers believed the line between patient input and external intimidation had been crossed; early data from the Sarepta muscular dystrophy drug's flawed development program stoked patient community expectations and made regulatory review difficult.

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